The clinical trial uniQure is conducting in HD uses a gene therapy known as AMT-130. This investigational treatment is the first one-time administered gene therapy to enter clinical testing for the treatment of HD. AMT-130 is administered once by neurosurgical procedure. There are two key components to AMT-130: a vector and a gene encoding a microRNA (miRNA). The vector acts as a delivery system and is based on a non-disease causing adeno-associated virus (AAV) that has been changed to carry and deliver a gene encoding a miRNA that will recognize, bind and non-selectively lower the human huntingtin protein. miRNA are small pieces of genetic material that can prevent production of a given protein. To “non-selectively lower” human huntingtin protein means that production of both the disease-causing mutant (mHTT) and normal huntingtin protein (HTT) will be decreased.
Under our U.S.-based protocol, we have completed enrollment of both the low-dose and high-dose cohorts (n=26) of the trial. We recently added a third cohort (n=18) that will explore an alternative surgical administration of AMT-130.